Global Orphan Drugs Market Forecast: 6.98% CAGR Growth by 2032, Driven by Rising Rare Disease Awareness and Favorable Regulatory Policies | DelveInsight

DelveInsight's Orphan Drugs Market Insights 2032 report delivers an in-depth evaluation of current and forecasted market trends, profiling key players, strategic alliances, and breakthrough drug developments. The report also examines the evolving regulatory environment, challenges surrounding pricing and reimbursement, and the competitive forces shaping the orphan drugs market outlook through 2032.

The global orphan drugs market is projected to witness significant growth over the forecast period, driven by rising awareness of rare diseases, increasing demand for innovative therapies, and supportive regulatory initiatives such as orphan drug designations and market exclusivity benefits. Designed specifically for rare conditions that impact a small portion of the population, orphan drugs are gaining prominence as governments, healthcare institutions, and pharmaceutical companies focus on addressing unmet clinical needs. Furthermore, advancements in genomics, enhanced diagnostic capabilities, and the efforts of patient advocacy groups are accelerating the research and development pipeline, paving the way for a new generation of targeted orphan therapies.

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Key Takeaways from the Orphan Drugs Market Report
• The global orphan drugs market was valued at USD 208.61 billion in 2024 and is expected to grow at a CAGR of 6.98%, reaching USD 357.26 billion by 2032.
• As per DelveInsight estimates, North America is anticipated to dominate the global orphan drugs market during the forecast period.
• Notable Orphan Drugs companies such as Sanofi, Biogen, Ionis Pharmaceuticals, Inc., Novartis AG, Alnylam Pharmaceuticals, Inc., F. Hoffmann-La Roche Ltd., AstraZeneca, Gilead Sciences, Inc., Johnson & Johnson Services, Inc., Mitsubishi Tanabe Pharma Corporation, BioMarin Pharmaceutical Inc., Vertex Pharmaceuticals Incorporated, Ultragenyx Pharmaceutical Inc., Kyowa Kirin Co., Ltd., Catalyst Pharmaceuticals, Inc., Chugai Pharmaceutical Co., Ltd., Sarepta Therapeutics, Inc., Astellas Pharma Inc., Pfizer Inc., Novo Nordisk A/S, and several others are currently operating in the Orphan Drugs market.
• In June 2025, Oncovita announced that the FDA granted Orphan Drug Designation to its lead investigational therapy, MVdeltaC, for the treatment of pleural mesothelioma, a rare and aggressive cancer with limited treatment options.
• In May 2025, Beam Therapeutics (Nasdaq: BEAM) announced the FDA granted orphan drug designation to BEAM-302, a liver-targeted lipid nanoparticle therapy using base editing to correct mutations in alpha-1 antitrypsin deficiency (AATD) patients.
• In May 2025, RELIEF THERAPEUTICS Holding SA announced that the FDA granted Rare Pediatric Disease designation to RLF-TD011 for treating epidermolysis bullosa (EB), a rare genetic skin disorder. The drug had already received Orphan Drug Designation for this indication.
• In May 2025, Mesoblast (ASX: MSB; Nasdaq: MESO) announced that the FDA granted seven years of orphan drug exclusivity for Ryoncil® (remestemcel-L) to treat steroid-refractory acute graft-versus-host disease (SR-aGvHD) in pediatric patients aged 2 months and older.
• In May 2025, Thermosome announced that the FDA granted Orphan Drug Designation to its lead compound THE001 for treating soft tissue sarcomas (STS), complementing an existing EMA designation and highlighting its clinical potential.
• In May 2025, Infinant Health announced that the FDA granted Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) to its investigational drug candidate, INF108, for the prevention of necrotizing enterocolitis in preterm infants.
• In April 2025, Esperion (NASDAQ: ESPR) announced it reached an agreement with the FDA to begin Phase III trials of bempedoic acid-both as monotherapy and in combination with ezetimibe-for pediatric patients with heterozygous and homozygous familial hypercholesterolemia (HeFH and HoFH). The trials are expected to launch this year, and bempedoic acid holds orphan drug designation for HoFH.
• In April 2025, NS Pharma announced that the FDA granted Orphan Drug Designation to NS-229, a selective JAK1 inhibitor being developed for eosinophilic granulomatosis with polyangiitis (EGPA). The designation supports NS-229's potential in regulating immune cell function to prevent tissue damage in this rare disease.
• In January 2025, Johnson & Johnson announced that its Nipocalimab Biologics License Application (BLA) received Priority Review designation from the FDA for the treatment of antibody-positive generalized myasthenia gravis (gMG) patients. The drug had previously been granted Orphan Drug Designation by both the FDA and European Medicines Agency (EMA).
• In January 2025, NMD Pharma A/S revealed that the FDA has granted Orphan Drug Designation (ODD) to NMD670, a novel oral small-molecule inhibitor targeting the skeletal muscle-specific chloride ion channel ClC-1, for the treatment of Charcot-Marie-Tooth disease (CMT).
• In January 2025, Elicera Therapeutics AB (publ), a clinical-stage cell and gene therapy company, announced that its drug candidate ELC-100 has received Orphan Drug Designation from the FDA for the treatment of pancreatic neuroendocrine tumors. This designation provides substantial regulatory benefits during the continued development and potential marketing approval of the treatment.
• In January 2025, KalVista Pharmaceuticals, Inc. reported that Japan's Ministry of Health, Labour and Welfare (MHLW) granted Sebetralstat Orphan Drug Designation for the on-demand treatment of hereditary angioedema (HAE) attacks in adults and adolescents aged 12 years and older.

For an in-depth look at the latest developments in the orphan drugs market, explore the key insights featured in DelveInsight's Global Orphan Drugs Market Report here: https://www.delveinsight.com/report-store/orphan-drugs-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=apr

Orphan Drugs Overview
Orphan drugs are pharmaceutical products developed specifically to diagnose, prevent, or treat rare diseases, also known as orphan diseases. In most regions, a rare disease is defined as one affecting a small percentage of the population - for instance, fewer than 200,000 people in the United States or fewer than 5 in 10,000 in the European Union. Due to the limited patient population, these conditions often lack sufficient market incentives for drug development without special regulatory support.

To encourage innovation, regulatory authorities such as the U.S. Food and Drug Administration (FDA), European Medicines Agency (EMA), and Japan's Pharmaceuticals and Medical Devices Agency (PMDA) offer benefits under orphan drug legislation. These include tax credits, reduced fees, market exclusivity, and grants for clinical research.

Orphan drugs span multiple therapeutic areas, including oncology, neurology, metabolic disorders, and genetic diseases. While they address significant unmet medical needs, their development is often challenged by high research costs, complex clinical trial designs, and small patient pools.

The global orphan drugs market is growing steadily, driven by advancements in biotechnology, increasing rare disease diagnosis, and supportive government policies. As innovation continues, orphan drugs remain vital in delivering life-changing therapies for patients with otherwise limited treatment options.

Orphan Drugs Market Insights

North America is projected to lead the orphan drugs market in 2024, driven by the growing prevalence of rare and genetic disorders, strong regulatory support, and an active pharmaceutical development pipeline. Legislative measures such as the U.S. Orphan Drug Act of 1983-which provides incentives like tax credits and market exclusivity-continue to foster innovation. The region's dominance is further supported by robust research and development, advancements in biotechnology, and a steady stream of new product launches.

According to the CDC (2024), approximately 35,000 individuals in the United States live with cystic fibrosis, while the Rare Disease Day Organization reports that 30 million Americans are affected by rare diseases, 80% of which are genetic in origin. Conditions such as spinal muscular atrophy (SMA), Batten disease, hemophilia, and Duchenne muscular dystrophy (DMD) underscore the urgent demand for new treatment options. In Canada, Batten disease occurs in roughly 1-35 births per 500,000.

Rising demand for targeted therapies is driving greater industry investment. In January 2025, Rigel Pharmaceuticals received FDA Orphan Drug Designation (ODD) for R289 in myelodysplastic syndromes, while Tempest Therapeutics secured ODD for amezalpat in hepatocellular carcinoma-highlighting North America's ongoing commitment to advancing therapies for underserved rare disease patients.

Learn more about the factors driving North America's leadership in the orphan drugs market by exploring the market outlook here: https://www.delveinsight.com/report-store/orphan-drugs-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=apr

Orphan Drugs Market Dynamics
The orphan drugs market is propelled by several key drivers. Rising awareness and diagnosis of rare diseases, coupled with advancements in genomics, biotechnology, and precision medicine, are enabling the development of highly targeted therapies. Supportive regulatory frameworks, such as the U.S. Orphan Drug Act and similar policies in the EU and Japan, provide incentives like tax credits, reduced fees, market exclusivity, and grants-encouraging pharmaceutical companies to invest in rare disease research. Increasing collaborations between biotech firms, academic institutions, and patient advocacy groups are also accelerating drug discovery and development. Additionally, the growing global rare disease patient pool and high unmet medical needs create significant market opportunities.

However, the market faces notable barriers. The small patient population for each rare disease limits commercial returns, often leading to high treatment costs that can restrict accessibility. Complex clinical trial designs, recruitment challenges, and lengthy approval processes further slow development timelines. Reimbursement hurdles, particularly in markets with stringent cost-effectiveness evaluations, can delay or limit patient access. Intellectual property disputes, manufacturing complexities-especially for biologics and gene therapies-and the risk of competition from emerging alternatives also pose challenges. Balancing innovation with affordability and timely access remains a critical factor for the sustainable growth of the orphan drugs market.

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Orphan Drugs Market Scope
• Coverage: Global
• Study Period: 2022-2032
• CAGR: ~6.98%
• Leading Companies: Sanofi, Biogen, Ionis Pharmaceuticals, Inc., Novartis AG, Alnylam Pharmaceuticals, Inc., F. Hoffmann-La Roche Ltd., AstraZeneca, Gilead Sciences, Inc., Johnson & Johnson Services, Inc., Mitsubishi Tanabe Pharma Corporation, BioMarin Pharmaceutical Inc., Vertex Pharmaceuticals Incorporated, Ultragenyx Pharmaceutical Inc., Kyowa Kirin Co., Ltd., Catalyst Pharmaceuticals, Inc., Chugai Pharmaceutical Co., Ltd., Sarepta Therapeutics, Inc., Astellas Pharma Inc., Pfizer Inc., Novo Nordisk A/S, and others.

Orphan Drugs Market Segmentation
• By Drug Type: Small Molecules, Biologics
• By Route of Administration: Oral, Parenteral
• By Indication: Oncology, Hematology, Ophthalmology, Neurology, Others
• By Distribution Channel: Hospital Pharmacies, Retail Pharmacies, Online Pharmacies
• By Geography: North America, Europe, Asia-Pacific, Rest of the World

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This release was published on openPR.